CRISPR: Medical breakthrough or too good to be true?

Cancer. Sickle cell anemia. AIDs. Muscular dystrophy. Cystic fibrosis. All five of these diseases induce debilitating physical, mental and emotional pain.

    Patients are typically subjected to frequent hospital visits and endless supplies of medication, yet at their core, what do these illnesses have in common?

    Gene mutations.

    Breast cancer patients have inherited gene mutations called BRCA1 or BRCA2. Gene mutations cause muscular dystrophy patients extreme weakness and muscle loss, while cystic fibrosis is the result of mutations in a gene that makes CFTR proteins. Currently, none of the diseases mentioned have cures, only treatments which can temper the side effects, causing sufferers to long for whatever gene they need or despise the one they don’t.

     That’s where CRISPR comes in.

    According to the CRISPR Therapeutics homepage, “CRISPR/Cas9 edits genes by precisely cutting DNA and then letting natural DNA repair processes to take over. The system consists of two parts: the Cas9 enzyme and a guide RNA.” With this gene-editing tool, genetic disorders and mutations could soon see huge breakthroughs and quite possibly cures, freeing victims from their medical imprisonment.

    But is it too good to be true?

What precisely is CRISPR?

“CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats.  It works with the Cas9 and Cpf 1 enzymes to cut genomic DNA and edit it,” Amie Mancine, Hoban biology and anatomy teacher who originally showcased this topic to her classes, explained. “Researchers have gotten very good at targeting specific sequences for CRISPR to edit, and so far it is fairly accurate.  Depending on the enzyme used, it can remove or edit damaged, mutated or diseased genes (Cas 9) or help to insert corrected, missing or new genes (Cpf 1).”

    Discovered in archaea and bacteria by Spanish scientist Francisco Mojica of the University of Alicante in 1993, CRISPR consists of repeating sequences of genetic code, interrupted by “spacer” sequences, which are transcribed into short RNA sequences. These sequences serve as genetic memories that help the cell detect and destroy invaders. With the help of Cas9 and Cpf 1, the sequence has the ability to edit, cut or insert genes, aiding in disorders caused by both missing and superfluous genes.

Medical Possibilities  

Though not publicly available yet, those who have tested CRISPR with Cas9 and Cpf 1 speak of its unlimited possibilities. Cures, stronger medicine and even disease elimination have all been theorized, illuminating the endless ways that CRISPR could save and prolong human life.

    “In terms of medicine, it could give us models for testing drugs and treatments, as well as possible treatments, preventions or cures to many single gene genetic disorders,” Mancine said.  “It may also allow us to prevent cancer and viral infections too. It’s uses are really endless —it could even be used in agriculture!”

    This discovery is especially important to Mancine, as her daughter Haley suffers from Phelan McDermid Syndrome (22q13 deletion syndrome). This deletion has left 11-year-old Haley developmentally delayed, nonverbal, wheelchair bound, legally blind and with many other symptoms, but with the help of her parents, two older brothers, multiple doctors and her trusty iPad, Haley lives surrounded with love and care.

    “Basically 29 genes are missing on the bottom of chromosome 22. I think this advancement could help her by giving her any of the 29 genes she is missing,” she stated. “It may already be too late to make much of a difference on her brain development since she is 11, but any improvement would give her a better quality of life.”

    Dr. Robert Glatter, a New York emergency room physician, recently wrote an article for Forbes magazine on the medical studies done by CRISPR researchers. He revealed that last year, biotech companies Juno Therapeutics and Kite Pharmaceuticals tested human T-cells engineered with CRISPR. The results showed remission in terminal leukemias.

    He also claimed that at Temple University, researchers eliminated HIV-1 from T-cells with CRISPR. Beyond the U.S., British scientists are utilizing CRISPR to eradicate malaria from the common mosquito bite and Chinese studies have corrected multiple lethal mutations, a great leap for disease study—if only human embryos had not be used, leading many to question the ethics of this research despite its results.

Ethical Setbacks

Embryonic stem cell research has been shamed by the Church since the experimentation began. As Church doctrine states that human life begins the moment of conception, anything unnatural implemented on the embryos is viewed as extremely unethical. Even In-Vitro Fertilization (IVF) is a disgraced practice, though it gives couples who suffer from infertility a child to love.

    This is where Mancine, a devout Catholic, draws the line.

    “The science is moving so fast, that the Church is having trouble keeping up. To me, every life is sacred and should not be used for research in this matter.  It doesn’t feel right to take a child’s life to give another child a better life,” she said. “As much as I would like to support it, I can’t morally do so. I am okay, however, with trying to make drought resistant plants to maintain a food supply for a future with a predicted shortage due to global warming.  No embryos used there.”

Regulation

With CRISPR, Cas9 and Cpf 1, the BRCA1 gene could be eliminated. Males with muscular dystrophy could finally have fully formed muscles, and the CFTR protein could transport water and ions like it’s meant to. But, if these genes could be edited, could parents have the power to change their child’s eye color from brown to blue? Their hair from black to blonde? Curly to straight?

    “Because the applications are endless, you could go to the extreme and ask how far will we go?” Mancine questioned. “Will we create a GATTACA-like society where we design our children?  Should we play God?”

    The possibilities are indeed endless, as Mancine and multiple researchers claim . But where should they be regulated? With all the good CRISPR can potentially cause, could the power it holds be taken advantage of? The possibility of saving human life from suffering is too strong to ignore, but regulations are necessary if we wish to honor the beauty of natural life—life that, with CRISPR and the proper regulations, could have the potential to become even more beautiful.